Aplastic anemia (AA), myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are rare diseases that result in bone marrow failure. When the marrow fails to produce blood cells, the resulting anemia, thrombocytopenia (low numbers of platelets in the bloodstream), and neutropenia (low numbers of neutrophils) lead to debilitating symptoms and death. Once considered distinct, these three diseases are now believed to be linked by similar pathophysiologies. Exploration of current research in AA, MDS, and PNH would greatly benefit from increased collaboration between basic and clinical scientists and scientists studying the individual diseases. Increased understanding of the molecular events driving these diseases and of the response to treatment are needed to define at-risk populations and improve current therapies. The Aplastic Anemia & MDS International Foundation, Inc. (AA&MDSIF) proposes to hold its fourth Bone Marrow Failure Disease Scientific Symposium on March 27-28, 2014, in Rockville, Maryland, with Richard M. Stone, M.D., from the Dana-Farber Cancer Institute, as Principal Investigator and Co-Chair of the Symposium and Neal S. Young, M.D., from the National Heart, Lung, and Blood Institute, as Co-Chair. The Symposium will bring together physicians treating these diseases and laboratory researchers studying the immunology and cell biology of bone marrow failure to discuss current areas of consensus and controversy, share recent research results, and propose recommendations for the highest priority directions for basic and clinical research needed to advance the field. The format will consist of six sessions: (1) Genetics and Genomics of Bone Marrow Failure, (2) Pathophysiology and New Molecular Targets in MDS, (3) Immunobiology of AA and PNH, (4) Transplant Treatments for Bone Marrow Failure, (5) Non-Transplant Therapy for AA, and (6) Non-Transplant Therapy of MDS. Two special sessions will address AA in the developing world and the future of bone marrow failure studies in the clinic and the laboratory. Each session will offer time for questions and discussions as well as for capturing recommendations for future research. The two days of the conference will also include poster and discussion sessions and networking opportunities. The conference planners will expand on previous efforts to attract young investigators to the Symposium (and to the bone marrow failure disease research field) by offering poster sessions, travel support, extended discussion opportunities with experts, and, if funds permit, a Young Investigators Prize. AA&MDSIF will publish lay versions of the proceedings, including research recommendations, on its website and meeting leaders will publish a summary of the proceedings for researchers and clinicians in a peer-reviewed journal.